Use of PTC124 for nonsense suppression therapy targeting BMP4 nonsense variants in vitro and the bmp4(st72) allele in zebrafish

Nonsense suppression therapy (NST) utilizes compounds such as PTC124 (Ataluren) to induce translational read-through of stop variants by promoting the insertion of near cognate, aminoacyl tRNAs that yield functional proteins. We used NST with PTC124 to determine if we could successfully rescue nonse...

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Detalles Bibliográficos
Autores principales: Krall, Max, Htun, Stephanie, Slavotinek, Anne
Formato: Online Artículo Texto
Lenguaje:English
Publicado: Public Library of Science 2019
Materias:
Research Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6481805/
https://www.ncbi.nlm.nih.gov/pubmed/31017898
http://dx.doi.org/10.1371/journal.pone.0212121

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6481805/
https://www.ncbi.nlm.nih.gov/pubmed/31017898
http://dx.doi.org/10.1371/journal.pone.0212121

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